Emapalumab for children with aplastic anemia
Official title: Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
Funded at Columbia University Health Sciences · NIH-11457555 · Data updated 2026-11-12
Doctors will give emapalumab to children with acquired aplastic anemia to try to stop the immune attack on the bone marrow and help it make blood cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | December 1, 2025 |
| Project end | June 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $650,000 |
| NIH project number | 7R01FD008175-03 |
| Project ID | NIH-11457555 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents with newly diagnosed acquired aplastic anemia who are within the initial diagnostic/treatment decision window and who can undergo infusions and frequent follow-up.
✕Less likely to help
Children with inherited bone marrow failure syndromes, those with severe organ dysfunction or uncontrolled infections, or those who have already completed immunosuppressive therapy or transplant are unlikely to benefit.
What this research studies
If your child is newly diagnosed with acquired aplastic anemia, they may be offered a short course of emapalumab during the initial decision period before other treatments start. The drug blocks a key immune signal (interferon gamma) thought to damage bone marrow, and it will be given by infusion with close monitoring of blood counts and infections. The project begins with a smaller safety phase and moves to a larger phase to see how many children show improved blood counts or need fewer transfusions. Standard options like immunosuppression or bone marrow transplant remain available if emapalumab does not lead to recovery.
Why it matters
Potential benefit: If successful, this treatment could reduce immune-driven bone marrow damage and help children recover blood counts, lowering transfusion needs and possibly avoiding or delaying transplant.
How similar studies have gone: Emapalumab has shown benefit in another immune condition (HLH), but using it for pediatric aplastic anemia is largely novel with limited prior data.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Oved, Joseph H — Columbia University Health Sciences
- Study coordinator: Oved, Joseph H
Related research
Other NIH-funded projects on the same topics.
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Clinical and immunologic study of pediatric severe aplastic anemia to personalize care
A pediatric-focused research effort will collect and study immune and clinical data to tailor treatments for children with severe aplastic anemia.
-
Aplastic Anemia Registry to Understand How Immune and Genetic Discoveries Affect Disease
A collaborative registry study to learn how genetic background and immune factors influence aplastic anemia and related bone marrow failure conditions in patients.
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Eltombopag: a new way to boost blood stem cells in aplastic anemia
A new drug approach aims to help people with idiopathic aplastic anemia by stimulating blood-forming stem cells through mechanisms that may not depend on traditional pathways.
-
Understanding how Eltrombopag helps with aplastic anemia
This research explores how the medication Eltrombopag helps patients with aplastic anemia by looking at its effects on blood-forming cells.
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Investigating a new treatment for pediatric aplastic anemia
This study is looking at how well a new treatment called emapalumab works for kids with acquired aplastic anemia, a rare blood disorder where the body stops making enough blood cells, to see if it can help them produce more blood cells and feel better.
-
Comparing bone marrow transplantation with immune therapy for severe aplastic anemia in young patients
This study is looking at whether bone marrow transplants from unrelated donors or immune suppression therapy works better for kids and young adults with newly diagnosed severe aplastic anemia, especially since not everyone has a sibling who can donate bone marrow.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.